On October 16, 2019 Eli Lilly and Company (NYSE: LLY) reported top-line results from its Phase 3 SEQUOIA trial evaluating pegilodecakin plus FOLFOX (folinic acid, 5-FU, oxaliplatin) compared to FOLFOX alone in patients with metastatic pancreatic cancer whose disease had progressed during or following a first-line gemcitabine-containing regimen (Press release, Eli Lilly, OCT 16, 2019, View Source [SID1234542298]). The SEQUOIA trial did not meet its primary endpoint of overall survival.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The most common Grade 3/4 adverse events occurring at a higher rate (>5% difference) on the pegilodecakin-plus-FOLFOX arm compared to the FOLFOX arm were neutropenia, thrombocytopenia, fatigue and anemia. Detailed efficacy and safety results will be submitted for presentation at a future medical meeting.

Metastatic pancreatic cancer is one of the deadliest major cancers, with just three percent of patients in the U.S. living five years after the cancer is diagnosed. In the U.S., pancreatic cancer is the third leading cause of cancer death and is expected to become the second leading cause of cancer-related death in the next decade.1 Globally, pancreatic cancer is the seventh leading cause of cancer-related death.2

"More than 56,700 Americans – mothers, daughters, fathers, sons, colleagues and friends – will be diagnosed with pancreatic cancer this year alone," said Julie Fleshman, JD, MBA, president and CEO of the Pancreatic Cancer Action Network (PanCAN). "Because this is an aggressive disease and the current scope of treatment options is limited, there remains an urgent need for meaningful solutions to improve outcomes for pancreatic cancer patients."

"Pancreatic cancer has proven to be one of the most difficult tumor types to treat and there have been very few recent treatment advancements in the later-line metastatic setting. We are grateful to the patients, investigators and researchers who participated in the study," said Maura Dickler, M.D., vice president, late phase development, Lilly Oncology. "While we are disappointed by the outcome of the SEQUOIA study, we look forward to the upcoming results in lung cancer, learning from those results and increasing our understanding of pegilodecakin’s novel mechanism of action in cancer immunotherapy."

Lilly gained pegilodecakin with the acquisition of ARMO BioSciences in June 2018. SEQUOIA was initiated by ARMO in March 2017 based on results of the Phase 1/1b IVY study, which evaluated pegilodecakin – used as a single agent and in combination with chemotherapy and with checkpoint inhibitor therapy – across multiple tumor types including pancreatic, non-small cell lung and renal cell cancers. Results from the IVY trial were also the basis for the ongoing Phase 2 CYPRESS 1 and CYPRESS 2 studies of pegilodecakin in combination with checkpoint inhibitors in non-small cell lung cancer (NSCLC). The CYPRESS studies were initiated by ARMO in March 2018 and results, expected in early 2020, will inform future studies of pegilodecakin in NSCLC. For the next stage of pegilodecakin’s clinical development, Lilly is focused on assessing biomarkers and conducting studies in NSCLC and other tumor types including renal cell carcinoma, where the molecule has shown promising activity.

Notes to Editors

About SEQUOIA
SEQUOIA is a global, multi-center, randomized Phase 3 study designed to compare the efficacy and safety of pegilodecakin in combination with FOLFOX (folinic acid, 5-FU, oxaliplatin) compared to FOLFOX alone in participants with metastatic adenocarcinoma of the pancreas whose disease had progressed on one prior gemcitabine-containing regimen. The primary endpoint of the study is overall survival and key secondary endpoints are progression-free survival and objective response rate. SEQUOIA was initiated by ARMO BioSciences in March 2017 and enrolled 567 patients.

About Pancreatic Cancer
Metastatic pancreatic cancer is one of the deadliest major cancers, with just three percent of patients in the U.S. living five years after the cancer is diagnosed. In the U.S., pancreatic cancer is the third leading cause of cancer death and is expected to become the second leading cause of cancer-related death in the next decade.1 Globally, pancreatic cancer is the seventh leading cause of cancer-related death.2

About Pegilodecakin
Pegilodecakin is an immunotherapy which stimulates the body’s natural defenses against cancer and expands tumor-attacking T cells. This class of T cells can infiltrate and destroy cancer cells. Pegilodecakin, a pegylated IL-10, has shown clinical activity as a single agent in renal cancer and promising results in combination with both chemotherapy and checkpoint inhibitor therapy across several tumor types including non-small cell lung cancer and renal cell carcinoma.

On October 16, 2019 Amarna Therapeutics, a privately held biotechnology company developing a next-generation SV40-based gene delivery vector platform named SVac that promises to transform gene-replacement and immunotherapy across many disease areas, reported that it has raised €10 million (Press release, amarna therapeutics, OCT 16, 2019, View Source [SID1234542314]). The round was led by Flerie Invest AB, a Swedish investment company with another substantial contribution coming from an innovation credit from the "Netherlands Enterprise Agency" (RVO.nl) and existing shareholder Pim Berger.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company plans to use this funding to progress development of its SVac platform towards a first in man clinical study to commence in two to three years from now.

In addition to raising new funds, Amarna has recruited a new Supervisory Board to help underpin this new ‘clinical’ phase of its growth and development. Thomas Eldered has been appointed as Supervisory Board Chairman. Bernhard Kirschbaum, Maarten de Chateau, Ted Fjällman, Pim Berger and Guillaume Jetten have also joined the Supervisory Board (see biographies at the end of the release).

Ben van Leent, CEO of Amarna said:
"We are very happy to have attracted such strong investors. This significant new funding allows Amarna to accelerate the development of SVac and our lead product AMA001, and to help us achieve our ultimate goal: to become a leading global gene therapy player".
"We would like to extend a warm welcome to all of the members of our new Supervisory Board, led by the outstanding healthcare and biotech pioneer Thomas Eldered. They bring many years of in-depth life science knowledge and entrepreneurship to Amarna".

Thomas Eldered, new Chairman of Amarna’s Supervisory Board, commented:
"I’m very much looking forward to working at Amarna. Its viral gene delivery vector platform has the potential to make major medical breakthroughs possible, so that patients can be actually cured of "significant" diseases for which, to date, effective treatment have not become available. Together with my highly qualified and experienced colleagues in our new Supervisory Board, I’m fully committed to help progress Amarna into the next important clinical stages of development of its groundbreaking technology".

SV40 Vectors are non-immunogenic in humans
Viral gene delivery vectors that are currently used for in vivo gene therapy are ineffective because the particles are instable upon injection (in the case of lentiviral vectors) or because the particles are immunogenic in humans (in the case of AAV vectors). Gene delivery vectors derived from the macaque polyomavirus Simian Virus 40 (SV40) are an attractive alternative to lentiviral and AAV vectors for clinical gene therapy. Humans can be considered naïve to SV40 since the virus only replicates in macaques, where it causes symptomless infections. Replication-defective SV40 vectors are non-immunogenic in humans and moreover, have the capacity to induce immune tolerance to the transgene products. SV40 vectors therefore hold a great potential for clinical applications treating genetic disorders, cancer, allergies and degenerative/inflammatory conditions such as neurodegenerative and psychiatric diseases, atherosclerotic cardiovascular disease, diabetes mellitus, arthritis, chronic obstructive pulmonary disease and many more.

Amarna’s SVac platform: the benefits
Amarna has genetically engineered the SV40 genome used for the production of vector particles and in parallel generated a novel Vero-based packaging cell line named SuperVero that produces similar numbers of vector particles to the currently used packaging cell lines but without contaminating wild type SV40 particles. Since SVac is safe, highly efficient, non-immunogenic in humans and vector particles can be cost effectively produced in SuperVero cells, Amarna’s vector platform paves the way to clinically evaluate a whole new generation of SVac-based therapeutics for today’s major diseases.

On October 16, 2019 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain (Press release, Intellia Therapeutics, OCT 16, 2019, View Source [SID1234542299]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Intellia’s data includes important updates about the company’s programs and platform development activities:

Oral Presentation:

"In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression"

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellia’s gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellia’s similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.
Abstract number: OR48
Session 5b: New delivery systems and technologies
Presentation date/time: Friday, October 25, 2019, 11:30 a.m. – 1:30 p.m. CET
Location: Room 113-115

Poster Presentations:

"In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics"

This poster presentation will highlight Intellia’s approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel O’Connell, Ph.D.
Poster ID Number: P655
Date: Wednesday, October 23, 2019

"Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia"

This poster presentation focuses on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San Raffaele
Poster ID Number: P111
Date: Wednesday, October 23, 2019

"Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout"

This poster presentation focuses on the company’s T cell engineering technology, which is being applied in its Wilms’ Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.
Poster ID Number: P162
Date: Thursday, October 24, 2019

"CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria"

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.
Poster ID Number: P552
Date: Thursday, October 24, 2019

On October 16, 2019 Exelixis, Inc. (Nasdaq: EXEL) reported that its third quarter 2019 financial results will be released on Wednesday, October 30, 2019 after the markets close (Press release, Exelixis, OCT 16, 2019, View Source [SID1234542315]). At 5:00 p.m. EDT / 2:00 p.m. PDT, Exelixis management will host a conference call and webcast to discuss the results and provide a general business update. Access to the event is available via the Internet from the company’s website.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 5489464 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on November 1, 2019. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 5489464. A webcast replay will also be archived on www.exelixis.com for one year.

On October 16, 2019 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized chimeric antigen receptor T cell ("CAR T") platform technology engineered to target patient-specific tumor neoantigens, reported that Jeffrey Eisenberg, Chief Executive Officer of Xenetic, will present at BIO Investor Forum on Wednesday, October 23, 2019 at 2:45 PM PT in San Francisco, CA (Press release, Xenetic Biosciences, OCT 16, 2019, View Source [SID1234542300]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As part of his presentation, Mr. Eisenberg will provide a Company overview and discuss the Company’s novel CAR T platform technology, called "XCART," a proximity-based screening platform capable of identifying CAR constructs that can target patient-specific tumor neoantigens, with a demonstrated proof of mechanism in B-cell Non-Hodgkin lymphomas. Xenetic is currently advancing the development program for XCART to confirm the positive preclinical results shown to date and to demonstrate a more attractive safety profile than existing therapies.

In addition to the presentation, management will also be available to participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference.

A live audio webcast of the presentation will be available on the IR Calendar in the Investors section of Xenetic’s website (www.xeneticbio.com) or by accessing the conference website here. Within three days of the event, a webcast replay will be made available on the Company’s website.

About BIO Investor Forum

Now in its 17th year, the BIO Investor Forum is an international biotech investor conference focused on early and established private companies as well as emerging public companies. The event features plenary sessions, business roundtables, therapeutic workshops, company presentations, and BIO One-on-One Partnering meetings. For more information, please visit the conference website here.